Gene Therapy Inquiry

Gene Therapy: A Promising Treatment for Alzheimer's Disease

Genetic mutation is an important factor that contributes to the pathogenesis and development of Alzheimer’s disease (AD). The genetic factors studied include the dominant mutations of genes encoding amyloid-β precursor protein (APP), presenilin 1 (PSEN1), and presenilin 2 (PSEN2). Additionally, more and more genes have been found to be potentially associated with AD, such as apolipoprotein E (APOE), glycogen synthase kinase 3 beta (GSK3B), dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A), and Tau. Although many studies on the treatment of AD have not been successful, gene therapy is still considered as a potential way against AD, and some gene-therapy-based therapeutics have entered the clinical trial stage. Existing strategies for gene therapy against AD include gene inactivation, genetic modification, and immunoregulation, etc.

About Alzheimacy

Alzheimacy fully understands that basic research to clarify the pathogenesis of AD and the development of new therapies for the disease are challenging and request long-term investment. But we work fearlessly. We do not give up even when challenged, pursuing innovation in all that we do. As a leading technology company in the life science field with extensive expertise, rich experience, and state-of-the-art facilities, Alzheimacy focuses on the development of gene therapy in AD, from molecular pathogenesis research to pre-clinical studies of drugs.

In the development of gene therapy in AD, our basic research focuses on exploring the pathogenesis of AD from the genetic level. Using our experience and expertise in biology and chemistry, we have established an integrated research platform that enables us to perform a variety of in vitro and in vivo experiments, including but not limited to gene expression profiling, genome-wide association study (GWAS), gene editing, gene silencing, biochemical experiments, neuroimaging techniques, AD transgenic animal models and iPSC-neuron. We believe that a powerful technology platform can help discover novel candidate genes related to AD and identify biological functions of candidate genes. Additionally, we are able to perform a comprehensive and systematic analysis of some AD-related candidate genes interactions in the context of AD using our computational biology platform.

The development of effective AD therapies is challenging, primarily due to the lack of reliable targets. We hope to utilize novel gene targets to start the development of gene therapy in AD and are actively identifying new possible gene targets for gene therapy.

In the strategy of introducing genes, efficient and safe gene therapy vectors are important for the success of gene therapy. Our delivery system technology platform supports the development and safety verification of gene therapy vector systems, which include viral vectors and non-viral vectors.

Small nucleic acid drugs, represented by small interfering RNA (siRNA) and antisense oligonucleotides (ASOs), have a bright future in gene therapy of neurological diseases due to their unique advantages. Our skillful technical team supports the design, synthesis and chemical modification of this class of drugs. In addition, our pharmacochemical scientists have experience in solving potential problems with small nucleic acid drugs, such as instability, insufficient affinity for target sequences, off-target/ toxic effects, and immune stimulation.

At Alzheimacy, we uphold the superior design principles and we understand the regulatory challenges that gene therapy developers face. We comply with all GLP, GMP and GCP guidelines, and maintain a Quality Management System (QMS).

If you are looking for a qualified supporter who can achieve your outstanding project on basic research or drug development in AD, please do not hesitate to contact us.